Memorial Sloan Kettering Cancer Center, Pediatrics, New York, NY, USA.
Haemophilia. 2010 Jul 1;16(4):625-31. doi: 10.1111/j.1365-2516.2010.02208.x. Epub 2010 Feb 10.
Cranial haemorrhage (CH) is a potentially serious complication in patients with severe congenital haemophilia with inhibitors (CHwI). Treatment includes bypassing agents, such as recombinant activated factor VII (rFVIIa). To examine the US experience in treating CH with rFVIIa, a retrospective review of the Hemophilia and Thrombosis Research Society 2004-2008 database was conducted. Among 29 patients with CHwI, 56 of the reported haemorrhages met the study criteria. Of these, 75% were traumatic and 80% were extracranial (ECH). The majority (8/11, 73%) of intracranial haemorrhages (ICHs) developed spontaneously. Conversely, most ECHs (39/45, 87%) followed trauma. ICHs were treated with a median/mean of 23/58 rFVIIa infusions over a median/mean of 7/9 days while ECHs were treated with a median/mean of 1/3 infusions (P = 0.011) over a median/mean of 1/1 day. The median/mean initial rFVIIa doses for all CHs were 106/137 microg kg(-1), and were similar for ICHs and ECHs. All ECHs were effectively controlled with rFVIIa; 44/45 bleeds were controlled within 24 h, one bleed was successfully treated perioperatively, and 27 ECHs required only a single dose. Nine out of 11 ICHs were effectively treated with rFVIIa; six ICHs were controlled within 24 h, one within 72 h and in two cases haemostasis was achieved during the perioperative period. No serious treatment-associated adverse events were reported. One patient died as a result of ICH despite the reported control of bleeding. In conclusion, standard dosing of rFVIIa was found to be safe and effective in treating CH with an efficacy rate of 100% for ECH and 82% for ICH.
颅内出血(CH)是患有严重先天性血友病伴抑制剂(CHwI)的患者潜在的严重并发症。治疗方法包括旁路制剂,如重组活化因子 VII(rFVIIa)。为了研究美国使用 rFVIIa 治疗 CH 的经验,对 2004-2008 年 Hemophilia and Thrombosis Research Society 的数据库进行了回顾性审查。在 29 名患有 CHwI 的患者中,报告的出血中有 56 例符合研究标准。其中,75%为创伤性,80%为颅外(ECH)。大多数(8/11,73%)颅内出血(ICH)自发性发生。相反,大多数 ECH(39/45,87%)继发于创伤。ICH 接受中位数/平均 23/58 次 rFVIIa 输注治疗,中位数/平均 7/9 天,而 ECH 接受中位数/平均 1/3 次输注(P=0.011),中位数/平均 1/1 天。所有 CH 的中位数/平均初始 rFVIIa 剂量为 106/137μg/kg,ICH 和 ECH 之间相似。所有 ECH 均经 rFVIIa 有效控制;44/45 例出血在 24 小时内得到控制,1 例在围手术期成功治疗,27 例 ECH 仅需单次剂量。11 例 ICH 中有 9 例经 rFVIIa 有效治疗;6 例 ICH 在 24 小时内得到控制,1 例在 72 小时内得到控制,在 2 例情况下,围手术期实现止血。未报告与治疗相关的严重不良事件。尽管报告了出血控制,但仍有 1 例患者因 ICH 死亡。总之,标准剂量 rFVIIa 治疗 CH 是安全有效的,ECH 的有效率为 100%,ICH 为 82%。